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Dive Brief:

Juno Therapeutics now expects to win U.S. approval for its experimental CAR-T therapy in acute lymphoblastic leukemia (ALL) as early as the first half of 2018, pushing back its timeline for potential approval from 2017, the company said on an investor call Thursday.

The Food and Drug Administration had halted Juno's trial for the therapy, known as JCAR-015, in early July after three patient died from cerebral edema. Juno linked the tragic fatal reactions to the addition of a chemotherapy agent to its pre-conditioning treatment regimen.

Juno suggested several modifications to the trial design to avoid the toxic effects and the FDA agreed, quickly lifting its clinical hold after a few days.

Dive Insight:

Juno had been aiming to be first on the market with a CAR-T therapy, racing against Kite Pharma and Novartis who are developing their own treatments. But the deadly reactions seen in its ROCKET trial and requisite modifications to its trial design will delay Juno's plans, company CEO Hans Bishop said Thursday.

Kite Pharma now looks set to be the first to file, eyeing this year for a submission to the FDA and an approval sometime in 2017, according to EP Vantage. Novartis appears more conservative and plans to file sometime in 2017 for its CTL-019 therapy.

Juno said its ROCKET trial is now open for enrollment with two key modifications. The chemo agent thought to be responsible for triggering the patient deaths, fludarabine, has been removed from pre-conditioning treatment. Juno will also restrict treatment to one patient per week until six new patients have received treatment.

"The process of getting IRB approval across multiple sites along with the gated enrollment for the next six patients leaves us to now estimate approval as early as the first half of 2018," Bishop said.

However Bishop did emphasize the company does not believe fludarabine alone caused the toxicity which lead to the patient deaths.

"We believe that the intensity of the two agent lymphodepletion combined with JCAR015 dose in the second of ALL contributed to some patients experiencing very rapid cell expansion which in turn correlates strongly with the risk of severe toxicity," explained Bishop.

This is important since Juno also uses fludarabine in the pre-conditioning regimen for another one of its CAR-T therapies, JCAR017.

Juno projects that therapy, aimed at non-Hodgkin lymphomas and B-cell malignancies, could see an approval from the FDA as early as 2018. Similar to JCAR015, JCAR017 also targets the CD19 protein on the surface of cancer cells.

Bishop said Juno hopes to have approvals for JCAR017 across four different blood cancers by as early as 2019.